Updates

September 23, 2020September 23, 2020
Amryt Plans Regulatory Submissions for Filsuvez Following Positive Phase 3 Data

Filsuvez, a topical formulation being developed by Amryt Pharma, significantly improved wound closure in people with epidermolysis bullosa (EB) taking part in a Phase ... Read more
September 17, 2020September 16, 2020
Interview With the Butterfly, Part 1

First in a series. My son Jonah was born with junctional epidermolysis bullosa in 2009. He is now 11 and in the sixth grade. I ... Read more
September 16, 2020September 22, 2020
Libtayo May Treat Advanced Skin Cancer in RDEB Patients, Case Report Shows

The immunotherapy Libtayo (cemiplimab-rwlc) may be a promising treatment for the skin cancers that sometimes appear in people with recessive dystrophic epidermolysis bullosa (RDEB) ... Read more
September 9, 2020September 9, 2020
Donor-derived Skin Grafts Feasible Way of Treating RDEB, Trial Reports

Transplanting skin grafts from healthy donors can be a safe and effective way of treating chronic wounds in people with recessive dystrophic epidermolysis bullosa, ... Read more
September 3, 2020September 15, 2020
Focusing on the Manna for Today

Our son Jonah is 11 years old now and just began the sixth grade. It blows my mind to think that it has been ... Read more

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January 23, 2018January 23, 2018

Factors Beyond COL7A1 Mutations May Contribute to Dystrophic EB Forms, Study Suggests

A study from Spain has raised the possibility that the development of dystrophic epidermolysis bullosa (DEB) could be attributable to additional factors besides mutations in the COL7A1 gene. Researchers reached this conclusion after studying three unrelated patients with different forms of ... Read more

Updates

Amryt Plans Regulatory Submissions for Filsuvez Following Positive Phase 3 Data

Interview With the Butterfly, Part 1

Libtayo May Treat Advanced Skin Cancer in RDEB Patients, Case Report Shows

Donor-derived Skin Grafts Feasible Way of Treating RDEB, Trial Reports

Focusing on the Manna for Today

Subscribe to our mailing list

Same But Different Photo Contest Celebrates People With Rare Diseases

Efficacy of Cream in Control Group May Explain ESSENCE Trial Failure, Study Reports

Rethink, Accept, and Appreciate Your Unique Self

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

If You Are Struggling, Find Your Army

Most EB Patients Want to Participate in Clinical Trials but Have Concerns, Survey Finds

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

New Streaming Channel Showcases Rare Disease Films

Scientists Identify 2 New COL7A1 Gene Mutations in Chinese Girl With RDEB

Choosing Between Bad and Worse, I Learned to Become an Advocate

Pivotal Phase 3 Trial Testing Topical Gene Therapy B-VEC Now Enrolling DEB Patients

My Butterfly Baby

Report Describes Successful Treatment Combo Against Itching in EB Pruriginosa

Abeona Restarts VIITAL Phase 3 Trial of EB-101, Presents Positive Phase 1/2 Data

IV Immunoglobulin May Help Treat Inflammation in Severe EBP, Case Report Suggests

SD-101 Cream Lacks Wound Healing Superiority in Phase 3 Trial, Study Reports

2 New Mutations Linked to Epidermolysis Bullosa Pruriginosa in Case Study

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

CRISPR/Cas9 Gene Editing May Correct Genetic Defect in DEB, Study Suggests

Coalition Will Address Racial Disparities in Rare Disease Communities

Collaboration Seeks Treatments for Diseases Caused by Nonsense Mutations

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

PTR-01 Shows Signs of Efficacy in RDEB Patients, Early Trial Data Reveal

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Factors Beyond COL7A1 Mutations May Contribute to Dystrophic EB Forms, Study Suggests

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Amryt Plans Regulatory Submissions for Filsuvez Following Positive Phase 3 Data

Interview With the Butterfly, Part 1

Libtayo May Treat Advanced Skin Cancer in RDEB Patients, Case Report Shows

Donor-derived Skin Grafts Feasible Way of Treating RDEB, Trial Reports

Focusing on the Manna for Today

Subscribe to our mailing list

Same But Different Photo Contest Celebrates People With Rare Diseases

Efficacy of Cream in Control Group May Explain ESSENCE Trial Failure, Study Reports

Rethink, Accept, and Appreciate Your Unique Self

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

If You Are Struggling, Find Your Army

Most EB Patients Want to Participate in Clinical Trials but Have Concerns, Survey Finds

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

New Streaming Channel Showcases Rare Disease Films

Scientists Identify 2 New COL7A1 Gene Mutations in Chinese Girl With RDEB

Choosing Between Bad and Worse, I Learned to Become an Advocate

Pivotal Phase 3 Trial Testing Topical Gene Therapy B-VEC Now Enrolling DEB Patients

My Butterfly Baby

Report Describes Successful Treatment Combo Against Itching in EB Pruriginosa

Abeona Restarts VIITAL Phase 3 Trial of EB-101, Presents Positive Phase 1/2 Data

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

IV Immunoglobulin May Help Treat Inflammation in Severe EBP, Case Report Suggests

SD-101 Cream Lacks Wound Healing Superiority in Phase 3 Trial, Study Reports

2 New Mutations Linked to Epidermolysis Bullosa Pruriginosa in Case Study

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

CRISPR/Cas9 Gene Editing May Correct Genetic Defect in DEB, Study Suggests

Coalition Will Address Racial Disparities in Rare Disease Communities

Collaboration Seeks Treatments for Diseases Caused by Nonsense Mutations

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

PTR-01 Shows Signs of Efficacy in RDEB Patients, Early Trial Data Reveal

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Pin It on Pinterest